Trial Outcomes & Findings for Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT NCT06578949)
NCT ID: NCT06578949
Last Updated: 2026-06-05
Results Overview
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
COMPLETED
PHASE3
18 participants
Baseline, Day 183 (Week 26)
2026-06-05
Participant Flow
The study included a 26-week Primary Treatment Period, and an additional 32-week Extension Treatment Period. The results for the Primary Treatment Period have been reported. Final analysis data will be reported after completion of the 32-week Extension Treatment Period.
Participant milestones
| Measure |
Ravulizumab
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Overall Study
STARTED
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18
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Overall Study
Received at Least 1 Dose of Study Drug
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18
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Overall Study
COMPLETED
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18
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Baseline characteristics by cohort
| Measure |
Ravulizumab
n=18 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Age, Continuous
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42.8 years
STANDARD_DEVIATION 10.4 • n=20 Participants
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Sex: Female, Male
Female
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3 Participants
n=20 Participants
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Sex: Female, Male
Male
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15 Participants
n=20 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=20 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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18 Participants
n=20 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=20 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=20 Participants
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Race (NIH/OMB)
Asian
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18 Participants
n=20 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=20 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=20 Participants
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Race (NIH/OMB)
White
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0 Participants
n=20 Participants
|
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Race (NIH/OMB)
More than one race
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0 Participants
n=20 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=20 Participants
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PRIMARY outcome
Timeframe: Baseline, Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
Outcome measures
| Measure |
Ravulizumab
n=17 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
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-81.3 percent change
Interval -84.2 to -78.3
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SECONDARY outcome
Timeframe: Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug.
Outcome measures
| Measure |
Ravulizumab
n=17 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26)
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94.1 percentage of participants
Interval 71.3 to 99.9
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SECONDARY outcome
Timeframe: Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183.
Outcome measures
| Measure |
Ravulizumab
n=18 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26)
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94.4 percentage of participants
Interval 72.7 to 99.9
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SECONDARY outcome
Timeframe: Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia \[hemoglobin \<10 g/dL\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN.
Outcome measures
| Measure |
Ravulizumab
n=18 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)
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0 percentage of participants
Interval 0.0 to 18.5
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SECONDARY outcome
Timeframe: Baseline, Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect.
Outcome measures
| Measure |
Ravulizumab
n=18 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)
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4.5 units on a scale
Interval 1.3 to 7.7
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SECONDARY outcome
Timeframe: Baseline, Day 183 (Week 26)Population: FAS included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect.
Outcome measures
| Measure |
Ravulizumab
n=18 Participants
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)
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26.6 g/liter (L)
Interval 13.8 to 39.3
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Adverse Events
Ravulizumab
Serious adverse events
| Measure |
Ravulizumab
n=18 participants at risk
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Hepatobiliary disorders
Liver injury
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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General disorders
Pyrexia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Other adverse events
| Measure |
Ravulizumab
n=18 participants at risk
Participants received a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks in Primary Treatment Period.
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|---|---|
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Investigations
Blood glucose increased
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Respiratory, thoracic and mediastinal disorders
Nasal congestion
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Gastrointestinal disorders
Chronic gastritis
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Gastrointestinal disorders
Diarrhoea
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16.7%
3/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Gastrointestinal disorders
Toothache
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Skin and subcutaneous tissue disorders
Eczema
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Skin and subcutaneous tissue disorders
Pruritus
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Musculoskeletal and connective tissue disorders
Arthralgia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Investigations
Electrocardiogram QT prolonged
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Musculoskeletal and connective tissue disorders
Pain in extremity
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Metabolism and nutrition disorders
Decreased appetite
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11.1%
2/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Metabolism and nutrition disorders
Hyperuricaemia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Metabolism and nutrition disorders
Hypoalbuminaemia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Metabolism and nutrition disorders
Hypokalaemia
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11.1%
2/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Psychiatric disorders
Insomnia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Nervous system disorders
Dizziness
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11.1%
2/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Nervous system disorders
Headache
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38.9%
7/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Eye disorders
Dry eye
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Ear and labyrinth disorders
Middle ear inflammation
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Cardiac disorders
Sinus bradycardia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Cardiac disorders
Tachycardia
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Respiratory, thoracic and mediastinal disorders
Cough
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Infections and infestations
Influenza
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Infections and infestations
Periodontitis
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Infections and infestations
Sinusitis
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Infections and infestations
Tinea pedis
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Infections and infestations
Upper respiratory tract infection
|
22.2%
4/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Investigations
Electrocardiogram ST-T segment abnormal
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Investigations
Weight decreased
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5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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|
Investigations
Weight increased
|
5.6%
1/18 • Baseline up to Week 26
Safety Set included all enrolled participants who received at least 1 dose (full or partial) of the study intervention.
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Additional Information
Alexion Pharmaceuticals Inc.
Alexion Pharmaceuticals Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place